Olutasidenib: from bench to bedside

The discovery of isocitrate dehydrogenase 1 (IDH1) mutations in acute myeloid leukemia (AML) and the resounding success of molecularly targeted therapies in related myeloid malignancies swiftly prompted the development of IDH1mut inhibitors. Olutasidenib (formerly known as FT-2102) is an orally administered novel IDH1mut inhibitor that entered clinical development in 2016, proceeded briskly through the developmental process, and was granted regular approval to treat patients with R/R IDH1mut AML on 1 December 2022. Single agent olutasidenib, a potent and selective IDH1mut inhibitor, demonstrated highly durable remission rates along with meaningful outcomes, such as transfusion independence, in patients with R/R IDH1mut AML. This review will examine the preclinical and clinical development and the positioning of olutasidenib in the IDH1mut AML treatment landscape.

Automated summary

The discovery of IDH1 mutations in AML and the success of molecularly targeted therapies led to the development of IDH1mut inhibitors. Olutasidenib, a novel IDH1mut inhibitor, showed promising results in treating patients with R/R IDH1mut AML, including durable remission rates and transfusion independence. This review examines the development and positioning of olutasidenib in the treatment of IDH1mut AML.